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Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension Studies.

Nieves Diaz1, Maria Juarez2, Caterina Cancrini3,4

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Journal of Immunology (Baltimore, Md. : 1950)
|October 29, 2020
PubMed
Summary
This summary is machine-generated.

Seletalisib, a PI3Kδ inhibitor, shows promise for activated PI3Kδ syndrome (APDS) by improving clinical and immunological features. This rare immunodeficiency treatment demonstrated a favorable risk-benefit profile over 96 weeks.

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Area of Science:

  • Immunology
  • Pharmacology
  • Genetics

Background:

  • Activated PI3Kδ syndrome (APDS) is a rare primary immunodeficiency caused by mutations in PI3K-related genes.
  • Limited therapeutic options exist for APDS patients, necessitating novel treatment strategies.

Purpose of the Study:

  • To evaluate the safety, tolerability, and efficacy of seletalisib, a selective PI3Kδ inhibitor, in patients with APDS1 and APDS2.
  • To assess the long-term effects and immunological changes associated with seletalisib treatment in APDS.

Main Methods:

  • A phase 1b study and subsequent extension study evaluated seletalisib (15-25 mg/d) in genetically confirmed APDS patients.
  • Primary endpoints included safety and tolerability; secondary endpoints explored clinical and immunological efficacy.
  • Seven APDS patients received seletalisib, with some continuing treatment for up to 96 weeks.

Main Results:

  • Seletalisib treatment led to improvements in lymphadenopathy, lung function, thrombocyte counts, and chronic enteropathy.
  • Immunological changes included decreased transitional B cells, increased naive B cells, and decreased senescent CD8 T cells.
  • Adverse events were generally manageable, with a sustained favorable risk-benefit profile observed up to 96 weeks.

Conclusions:

  • Seletalisib demonstrated clinical and immunological benefits in patients with APDS.
  • The PI3Kδ inhibitor offers a potential new therapeutic avenue for this rare immunodeficiency.
  • Long-term treatment with seletalisib appears safe and effective for managing APDS.